COMPOUNDS TARGETING PAX3::FOXO1 FUSION PROTEIN
Application
US20260083724A1
Kind: A1
Mar 26, 2026
Assignee
Georgetown University
Inventors
Aykut Üren, Jeffrey A. Toretsky
Abstract
Compounds comprising a PAX3::FOXO1 fusion protein binding moiety, a bivalent linker, and an E3 ubiquitin ligase binding moiety. The compounds can be used to reduce PAX3::FOXO1 protein levels in a subject with fusion-positive rhabdomyosarcoma (FP-RMS), to ubiquitinate a PAX3::FOXO1 fusion protein in a cell, to treat FP-RMS in a subject in need thereof, and/or to treat a disease caused by the overexpression of PAX3::FOXO1 protein in a subject in need thereof.
CPC Classifications
A61K 31/4709
A61P 35/00
Filing Date
2025-09-26
Application No.
19341825